Revision 1 issued: September 5, 2019

This guidance document is intended to assist applicants in the preparation and submission of Humanitarian Use Device (HUD) designation requests to the U.S. Food and Drug Administration’s (FDA or Agency) Office of Orphan Products Development (OOPD). It is also designed to assist FDA reviewers in their evaluation and analysis of HUD designation requests (“HUD requests” or “requests”). Topics addressed in this guidance include:

• demonstrating in HUD requests that the device is designed to treat or diagnose a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year;
• how this demonstration varies depending on whether the device is intended for therapeutic or diagnostic purposes;
• how properties of the device may affect this demonstration; and
• for the purpose of a HUD request, identifying a medically plausible subset (“orphan subset”) of persons with a given disease or condition that affects or is manifested in more than 8,000 individuals in the United States per year.

This guidance addresses only HUD requests, which are the first step in seeking marketing approval of a HUD. This guidance does not address the second step in this marketing approval process—namely, the submission of a Humanitarian Device Exemption (HDE) application to the Center for Devices and Radiological Health (CDRH) or to the Center for Biologics Evaluation and Research (CBER). For more information on the preparation and submission of HDE applications, see Humanitarian Device Exemption (HDE) Program: Guidance for Industry and Food and Drug Administration Staff.

This guidance is responsive to the congressional mandate in section 740 of the fiscal year 2010 U.S. Appropriations Act (Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations Act, 2010, Public Law 111-80) dated October 21, 2009. Among other things, the congressional mandate required that the Commissioner of Food and Drugs establish a review group within FDA to describe its findings and make recommendations on issues related to rare and neglected diseases and, in part, to develop guidance document(s) based upon these recommendations.

In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.